The Senate Health, Education, Labor and Pensions (HELP) Committee seeks to begin a process to address the status quo in the development of new drugs and medical devices and identify how Congress can improve policies and promote efficiency and effectiveness in such development, according to a report by the Committee titled Innovation for Healthier Americans. The report states that the Committee also hopes to pass “transformational legislation” for President Obama to sign later this year.

Background

The Committee’s report stresses, “It has never been more difficult to bring a therapy through the development pipeline,” and notes that it costs between $1 billion and $2 billion to have a drug approved by the FDA. In addition to monetary cost, the lengthy approval process slows down the release of one product, while keeping resources, researchers, doctors, and providers from moving on to new treatments and investigational therapies. The venture capital community is losing interest in drugs and devices in the U.S. because of “increasing regulatory burden and uncertainty” and is shifting its focus to Europe and Asia. Because of these issues, the Committee seeks to gain insight on the allocation of resources and the oversight of development.

Funding for Basic Research

The National Institutes of Health (NIH) is vital in the support of basic research, representing one-fifth of federal research and development spending. More than 80 percent of NIH funding is invested in extramural research, which is awarded through the peer review process, and about 11 percent is spent on intramural research. Academic research institutions rely largely on federal grants (60 percent), which, the report states, “leaves an enormous capacity for growth in support from non-government entities and opportunities for far greater partnership and collaboration between academic research institutions, industry, patient groups, and other stakeholders.” According to the Committee, finding ways to facilitate enhanced partnership between government and non-government entities to support research is critical to advancing medical innovation.

Advancing Research to Clinical Testing

Despite the large number of promising candidates for new drugs, many are unable to move beyond animal studies to clinical testing, as scientific and economic challenges can be too great to justify further investment and study until it is ascertained that the candidates are low-risk enough to develop with limited resources. Even when drugs to get to clinical testing, the report states that 80 percent that make it to human trials are never approved and never commercially available to patients because they are deemed to be toxic. To address these barriers, the Committee recommends improving predictive capabilities for toxicology and efficacy, as well as use of disease registries to more easily—and more affordably—enroll eligible patients in clinical trials.

Improving Clinical Trials

Clinical trials face challenges such as “spiraling costs, high failure rates, administrative inefficiencies, the rise of precision medicine, and regulatory hurdles.” The Committee notes that the current approach to clinical trials leads to administrative inefficiencies, increasing the time and cost involved, but that advancements are being made to define data standards and streamline clinical trials. The NIH also supports clinical trial networks that increase patient engagement and involvement with clinical trials. The report states, “[W]e can see the promise of more efficient clinical trials, but the promise has not yet been realized. Currently, efforts are duplicated, best practices are not shared, and transformative innovations are not scaled up.” The Committee also notes that regulatory barriers—such as the FDA requirement of three-phase clinical trials—prevent clinical trials from being efficient and streamlined and suggests that a more flexible and responsible approach could reduce costs in terms of time and money, allowing drugs to reach patients more quickly. Similar streamlining for medical device review is also suggested.

The gold standards for approval. The standards for approving drugs and medical devices are decades old, the report states, and expediting medical product review is not a new concept. Drugs and biologics can qualify for expedited designations or pathways toward approval, such as: (1) accelerated approval pathways for drugs treating a serious condition and providing a meaningful advantage over other therapies if they are demonstrated to be reasonably likely to provide clinical benefit; (2) fast track designation to expedite development and allow for rolling review of drugs treating a serious condition, if data demonstrates the potential to address an unmet medical need; (3) priority review for drugs treating a serious condition and providing a significant improvement in safety and effectiveness; and (4) breakthrough therapy designation for drugs intended to treat serious conditions with preliminary clinical evidence that the drug may demonstrate a substantial improvement on a clinically significant endpoint over available therapies.

The FDA released a guidance on a pilot project allowing devices treating or diagnosing a life-threatening or debilitating disease to have early and often interaction with the FDA to shorten approval time, but “[t]he impact of this guidance and pilot program remain to be seen,” according to the report.

Global Competition in Product Development

Despite the U.S.’s long-held position as the predominant global player in drug and device development, competition is growing. The Committee states that legislators and innovators “have a critical role in ensuring that the U.S. maintains superiority in medical product development and that American patients get the best treatment possible.” Globalization results in more drugs and devices being manufactured beyond U.S. borders, creating additional challenges for regulators in protecting the public’s health. Regulatory harmonization presents an opportunity to reduce international drug development costs by streamlining and limiting the requirements that companies must fulfill to market a drug or device globally. However, current policy deviates from this goal, as the FDA participates and supports an international standard, “only to then raise the bar in its draft guidance.” The Committee states that it is a goal to align FDA policies with international standards.